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Novel gene variants in Polish patients with Leber congenital amaurosis (LCA)

BACKGROUND: Leber congenital amaurosis (LCA) is a rare retinal disease that is the most frequent cause of congenital blindness in children and the most severe form of inherited retinal dystrophies. To date, 25 genes have been implicated in the pathogenesis of LCA. As gene therapy is becoming availab...

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Podrobná bibliografie
Vydáno v:Orphanet J Rare Dis
Hlavní autoři: Skorczyk-Werner, Anna, Niedziela, Zuzanna, Stopa, Marcin, Krawczyński, Maciej Robert
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2020
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7731562/
https://ncbi.nlm.nih.gov/pubmed/33308271
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01634-y
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